New Funding and Approval Pathways Prove Popular

Compared to other manufacturing and technology industries, the bio/pharmaceutical industry has one of the longest product development timelines. The research, development, testing, and regulatory review process typically extends for more than a decade, often exhausting the financial resources and commitment of investors. For patients with unmet medical needs, the wait for an effective therapy can be long, frustrating, and even life threatening. Late-stage failures of promising drugs can be devastating to the developer and patient alike.

Bio/pharmaceutical companies continue to explore different research, collaboration, and funding strategies in the search for ways to accelerate drug development. Assistance may be coming from an unlikely source: US Congress.

The 16-day shutdown of the US government demonstrated the divisiveness and dysfunction that has characterized Congress. More than half of all FDA employees were furloughed; research programs stalled at National Institutes of Health; and investigators at the Centers for Disease Control were not available to track outbreaks of infectious diseases. However, two pieces of legislation that took effect in 2012 are showing signs of having a positive impact on bio/pharmaceutical development, encouraging business growth, and hopefully resulting in improved patient access to effective drugs.

The Jumpstart Our Business Startups (JOBS) Act, signed into law in April 2012, gives emerging growth companies an easier path to raise capital privately or to seek public financing. It also reduces financial regulatory and compliance burdens on certain new public companies, enabling them to focus more on scientific research and less on financial reporting. The Biotechnology Industry Organization (BIO) reports that more than 40 biotech companies have gone public using provisions available through the JOBS Act (1).

The Food and Drug Administration Safety and Innovation Act (FDASIA), which included reauthorization of the Prescription Drug User Fee Act through September 2017, also provided for the timely review of new drug and biologic license applications. Since the legislation took effect in October 2012, the Pharmaceutical Research and Manufacturers Association, BIO, and drug companies have commended FDA for improved communication and collaboration with sponsors. FDA’s breakthrough therapy designation was developed to expedite the development and review of drugs for serious or life-threatening conditions and provide more intensive FDA guidance on an efficient drug-development program. Other expedited programs are fast track designation, accelerated approval, and priority review. Although no drugs have been approved under the new designations, the pathways are proving to be popular.

In fiscal year 2013, the Center for Drug Evaluation and Research received 92 breakthrough requests, approved 27, and denied 41; the remaining requests are pending approval. For almost 98% of the requests, action was taken within 60 days of receipt of the request. The Center for Biologics Evaluation and Research denied 8 of the 10 requests it received through Aug. 31, 2013 (2).

Both the JOBS Act and FDASIA passed with bipartisan support. In a few months, as members of Congress engage in the next round of budget and debt-ceiling debates, perhaps they should take a lesson from these bipartisan efforts and reach a consensus that makes sense for the nation as a whole.